FDA approves first new ‘breakthrough’ treatment for severe depression in decades—What is esketamine?

This story has been updated.

The United States Food and Drug Administration on Tuesday approved the first new treatment for depression in 30 years, a nasal spray that can relieve severe symptoms in hours instead of weeks.

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For more than a decade, scientists have been pointing to the 1980s "club drug" ketamine as a possible solution for treatment-resistant individuals. And last month, an expert panel recommended the FDA approve a new nasal spray with the anesthetic's active ingredients.  The treatment, called esketamine, will be marketed under the name Spravato.

"This is potentially a game-changer for millions of people," Dennis Charney, dean of the Icahn School of Medicine at New York's Mount Sinai, told NPR.

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The expert FDA panel, which featured the Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee, voted the benefits of Janssen Pharmaceutical Companies' fast-acting esketamine 28-mg single-use nasal spray outweighed the risks.

"The committees based their support on the safety and efficacy data from five Phase 3 studies in patients with treatment-resistant depression: three short-term studies; one maintenance of effect study; and one long-term safety study, according to a Janssen Pharmaceutical Companies press release last month. And any adverse effects (increased blood pressure, dizziness, dissociative symptoms) "occurred shortly after dosing...were transient...and resolved the same day."

» RELATED: Is it safe to take ketamine for severe depression?

"The thing I'm most concerned about really is diversion and misuse and things like that," temporary FDA voting member Lee Hoffer of Case Western Reserve University told Medscape. Hoffer has urged the agency to keep a close eye on safety, which they plan to continue doing through their Risk Evaluation and Mitigation Strategy (REMS).

According to FDA’s Tiffany Farchione, “the drug will only be available through a restricted distribution system and it must be administered in a certified medical office where the health care provider can monitor the patient” to mitigate potential misuse and abuse. Additionally, “because of the risk of sedation and dissociation, patients must be monitored by a health care provider for at least two hours after receiving their Spravato dose.”

One in six adults will have depression at some time in their life, according to the Atlanta-based Centers for Disease Control and Prevention. More than 300 million people of all ages suffer from the mental disorder, and it's the leading cause of disability worldwide.

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While treatments such as antidepressants and psychotherapy exist, those with severe, treatment-resistant depression often involving suicidal thoughts may find such options are ineffective.

Esketamine’s fast-acting role spurred by ketamine’s active ingredients is what makes this possible treatment for severe depression most useful, experts say. Unlike other commonly used treatments, which can take several weeks to begin working, esketamine can offer symptom relief more quickly, potentially lowering one’s risk of self-injury or suicide.

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But some experts are wary. According to Medscape, it's still unclear what about ketamine's biology triggers an antidepressant response in one day when most antidepressants usually need two weeks or longer to make an impact. And what does ketamine do to the brain that antidepressants don't do?

Still, "well over 3,000" patients in the U.S. and Canada have been treated with ketamine so far, Yale University psychiatry professor Gerard Sanacora told NPR in 2017.

When other doctors ask him how he can offer the drug to patients with limited information about its effects, Sanacora responds with this answer: "If you have patients that are likely to seriously injure themselves or kill themselves within a short period of time, and they've tried the standard treatments, how do you not offer this treatment?"

When depressed patients who have exhausted their options take ketamine, Sanacora said, 50-75 percent of them feel at least 50 percent better within one day.

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According to the New York Times, despite federal approval, clinics all over the country have began administering intravenous anesthetic ketamine for depression "as a series of treatments, over a period of days or weeks, and sometimes including follow-up or 'booster' visits months later." Such "off-label" treatments average $3,000 and usually aren't covered by insurance, nor have their effectiveness been thoroughly studied.

But now that it's approved, ketamine's "chemical sibling" esketamine, which will be covered by most insurers, is the first new depression treatment in decades. As for how much it'll cost, "treatment will be between $2,360 and $3,540," Janssen executives told The New York Times. "And experts said it will give the company a foothold in the $12 billion global antidepressant market, where most drugs now are generic."

Read the new FDA press release at FDA.gov.